• Treatment of Ataxia - Genetics Based

    Explore cutting-edge genetic therapies for brain disorders, with a focus on antisense oligonucleotides (ASOs). This presentation details ASO mechanisms, preclinical development for Spinocerebellar Ataxia type 3 (SCA3), and promising ASO approaches for SCA1. Discover how ASOs target RNA and DNA, their efficient brain cell uptake, and ongoing challenges in widespread delivery.

    Ronald Buijsen Speaker: Ronald Buijsen

    AdvancedLevel: Advanced

    2026Publication year: 2026

    Topics in this course:

    ERN RND ERN-RND Post-graduate curriculum ataxias ataxia Genetic Therapy Antisense Oligonucleotides BrainDisorders Neuroscience SCA